Expression and Processing of Mature Human Frataxin after Gene Therapy in Mice

Researchers assessed mature exogenous hFXN expression levels in the heart and liver of C57Bl/6 mice seven-ten months after intravenous administration of a recombinant adeno-associated virus encoding hFXN (AAVrh.10hFXN) and examined the potential for hFXN truncation in mice.
[Scientific Reports]
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